2020

The European Patients’ AcademyEUPATI

What this presentation covers:

Why EUPATI?

Health research & policy is changingat a fast pace

Innovation transforms the lives of patients with serious, life-long conditions:

Molecular targets/pathways Genome sequencing, Translational research Personalized medicine

• Small trial populations • Biomarkers, companion diagnostics

Need for post-marketing data Health Technology Assessment,

QoL, endpoints, comparators BUT long term pressure on health budgets

– here to stay

Window ofopportunity trial design relationship

between researchers, regulators, industry, patients

4

Public

Research Ethics Committees

Competent authorities

Policy makers/Research Policy

HTA agencies& committees

Research subject

Info provider

Advisor

Reviewer

Co-researcher

Driving force

Clinical Research

Patients have a key role in all aspects ofhealth-related research

Trial protocol design, informed consent, ethical

review, marketing authorization, value

assessment, health policy

Patient involvement in practicewithin the R&D life cycleSource: Geissler, Ryll, Leto, Uhlenhopp – www.eupati.eu

Source: Geissler, Ryll, Uhlenhopp, Leto (2016): www.eupati.eu

Public

Research Ethics Committees

Competent authorities

Policy makers/Research Policy

HTA agencies& committees

Research subject

Info provider

Advisor

Reviewer

Co-researcher

Driving force

Clinical Research

Patients have a key role in all aspects ofhealth-related research

Trial protocol design, informed consent, ethical

review, marketing authorization, value

assessment, health policy

What is EUPATI?

European Patients’ Academy:Paradigm shift in empowering patients on medicines R&D Launched February 2012 as a public private

partnership, 33 consortium members,Funded by Innovative Medicines Initiative

Has developed and disseminatedobjective, credible, correct and up-to-date public knowledge about medicines R&D

Is building competencies & expert capacity among patients and the public

Has been a game changer and driver of patient engagement in R&D

Continues as a permanent educational program led by the EPF (since March 2017)

EUPATI Patient ExpertsTraining Course-- for expert patients

EUPATI develops education targeted at different levels

EUPATI Educational Toolbox-- for patient advocates

EUPATI Internet Library-- for the health-interested public

English FrenchGermanSpanishPolishItalian RussianDutchDanish(Portuguese)(Romanian)

English

1.Discovery of Medicines

2.Pre-clinical Development

3.Clinical Development

4.Clinical Trials 5.Regulatory

Affairs, Drug Safety, Pharmaco-vigilance

6.Health Technology Assessment

Reflecting European diversity: 9 languages

9 languages:• English • French• German• Spanish• Polish• Italian • Russian• Dutch• Danish

Many European countries:

UK, Ireland, Malta, France, Luxemburg, the francophone Belgium, Germany, Austria, Switzerland, Spain, Italy and Poland, plus Russian-speaking population in Central and Eastern Europe

Plus additional countries and languages in preparation by the community, e.g. Portuguese,Romanian etc.

Unique and unprecedented partnership between patient organisations, other public partners and pharmaceutical industry based on the philosophy of the IMI

• Unique in IMI that EUPATI is patient- and not industry-led

• Opportunity to establish an effective, transparent and credible partnership

• Exceptional learning experience for industry representatives to work with patient organisations in a constructive and reflective manner, which could serve as a role model in other environments as well

• Making best use of industry expertise in medicines R&D,incl. e.g. the legislative environment and development of ITP as required by law

Opportunity to strengthen the voice of the patients in the search for innovation and new medicines

Chance to reduce fear, lack of trust and misconception among patients regarding the pharmaceutical industry

Uniqueness of the partnership

What we bring in and what we produce will be owned by the public

All EUPATI material has been provided under the"Creative Commons License"• Content can be copied, distributed, edited, remixed, and

built upon, all within the boundaries of copyright law on non-commercial basis

• Authorship and licensing needs to stay intact and mentioned on all derivatives

• Similarly applied by e.g. Wikipedia, Google, and many others

See http://creativecommons.org/licenses/by/3.0/

Public license model guaranteesownership and re-use by the public

What has EUPATI delivered?

Facts and numbers (May 2020)

13,545

3,700,000

250,958

232

152

432,089 (613)

13 8

– users on busiest day in April 2020

– total users since launch

– European users of most used language

EUPATI trainees graduated from Cohorts 1, 2, 3 –

–words in the Toolbox (number of content pieces)

- 6 ENP run webinars- 7 general webinars

– new sessions developed for Cohort 3 Event 1

– countries use the Toolbox

EUPATI‘s work was based on solid research on patient & public attitudes towards medicines R&D

Review of existing information resources;230 resources reviewed.

Review of research literature; 12600 titlesand abstracts; 134 studies included

Public survey; 6931 responses in GB, Spain, Poland, Italy, France and Germany

Qualitative research with patients and thepublic; n=125 people

Qualitative research with stakeholders; n=56 (incl. policy makers, pharmaceuticalindustry, clinical research professionals)

Patient advocate survey; 468 responsesfrom across Europe

Patient advocate survey focused on course delivery; 41 responses

Results published in BMJ Open:

The EUPATI Patient Expert Training Course

EUPATI Patient Expert Training Courses

+Online self-learning

250 hours of e-learning and 8 days for two Face-to-Face meetings

over a period of 14 months

2 Face-to-face trainingweeks

EUPATI Patient Expert Training Courses

14-month in-depth training course of patient advocates on medicines R&D

152 graduates from 37 countries and 80+ disease areas completed Course 1, 2 & 3

Involvement of EUPATI Fellows and trainees evidenced in R&D projects, regulators, ethics

EUPATI Patient Expert Training Course: Rollout of course modules

EUPATI Courses 1, 2 & 3 Trainees by Disease Areas (74)2Achondroplasia1Adrenoleukodystrophy1Allergy and airways diseases3Arthritis

1Asperger´s syndrome/Fibromyalge and Chronic Fatigue Syndrome

1Asthma1Attention Deficit Hyperactivity Disorder1Autoimmune disease1Autoinflammatory diseases1Brain tumours5Breast cancer7Cancer2Cardiovascular Diseases1Chronic Fatigue2Chronic myelogenous leukemia1Chronic Pain1Coeliacy1Congenital Disorder of Glycosylation3Cystic Fibrosis3Diabetes1Disability1Ehlers-Danlos Syndrome1Endocrine tumours2Endometriosis1Epidermolysis bullosa (EB)1Fibromyalgia2Gaucher Disease4General advocacy1Genetic Disorders3Haemophilia

10Hematological Cancer1Hereditary haemorrhagic telangiectasia (HHT)5HIV1Incontinent patients1Leber’s Hereditary Optic Neuropathy3Leukemian (incl Chronic myelogenous leukemia)1Lipoprotein Lipase Deficiency

2Lung conditions4Lupus1McCune Albright syndrome5Melanoma1Mental Health5Multiple Sclerosis2Muscular atrophy6Muscular Dystrophy1Myeloma1Neurological Diseases1Neurofybromatosis1Oral Cancer2Paediatric Diseases2Paediatric Cancer2Parkinson's Disease2Pituitary disorders1Preterm Birth1Primary Immunodeficiences3Liver diseases (incl Primary Sclerosing Cholangitis)1Prostate Cancer1Pulmonary Hypertension

10Rare Diseases1Rare diseases/Friedrich’s Ataxia

1Rare diseases/Paroxysmal nocturnal hemoglobinuria

1Rare diseases1Rare diseases/Tuberous Sclerosis1Respiratory diseases1Rett Syndrome8Rheumatic diseases1Sanfilippo Syndrome2Sight loss1Spinal Cord Injury2Spinal Muscular Atrophy2Thalassaemia1Thalidomide victims1Tourette syndrome1Vasculitis

EUPATI courses 1, 2 & 3 trainees:36 countries

1Albania3Austria9Belgium1Croatina2Cyprus4Denmark2Finland5France1Georgia6Germany9Greece4Hungary

11Ireland1Israel7Italy1Kyrgyzstan1Latvia2Lithuania

1Luxembourg2Macedonia3Malta8Netherlands3Norway3Poland8Portugal6Romania3Russian Federation2Serbia2Slovakia1Slovenia

10Spain1Sweden9Switzerland1Turkey1Ukraine

21United Kingdom

Specific activities and roles developed by Fellows of the Course

“I am more respected as a EUPATI fellow…”

Advisoryrole

Promoted in roles

Involved in training

Involved in policy/

advocacy

Speaking jobs

Involved in R+D

Knowledge multiplier

Better negotiator

Formalised roles

Empowerment = respect = meaningful involvement = pride

Roles change as a result of the course, creating patient leadersRole Before EUPATI After

Member of patient organisation, not actively involved 17% 2%Active role in a patient organisation 62% 71%Leadership role in a patient organisation 62% 71%Employee of a patient organisation 25% 23%Volunteer role in a patient organisation 60% 67%Presenting at conferences, workshops etc. 63% 83%Advising a pharmaceutical company 13% 44%Advising a regulatory agency 21% 42%Advising a reimbursement agency 4% 8%

EUPATI Fellows are increasingly taking leadership roles and are engaging with pharma, regulators and HTA bodies.

Role changes also imply identity shifts.

What our EUPATI trainees say about the EUPATI Patient Expert Training Course…

25

Šarūnas Narbutas from Lithuania (Lithuanian Cancer Patient Coalition, POLA):“10 days of superbly engaging discussions with 50 capable patient advocates is the capacity building experience equal to none”.

Begonya Nafría from Spain(Hospital Sant Joan de Déu, Barcelona):“It´s essential that patients and their families are involved in research on rare diseases. They have an immense expertise, and their needs must be taken into account”.

What our EUPATI trainees say about the EUPATI Patient Expert Training Course…

26

Birgit Bauer from Germany(MS patient and blogger about Multiple Sclerosis):“(…) I use my gained knowledge for articles, blogposts and for talks. I now have better arguments in discussion panels and understand the different perspectives of different stakeholders.”

Maria Piggin from England(PNH Support):“I have been provided with tools which give me confidence to assert the patients' voice. Each process examined highlights how a patient advocate can add value.”

What our EUPATI trainees say about the EUPATI Patient Expert Training Course…

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Margaret Graham McDonald from Scotland(Healthcare Improvement Scotland):“The EUPATI course has harnessed the skills I needed to play an effective and active part in Research & Development with confidence.”

Stefano Mazzariol from Italy(Parent Project Onlus):“I would like to work with stakeholders to bring patients closer to research. I wish that all patients could benefit from the knowledge that I am acquiring in this course.”

The EUPATINational Platforms

21 EUPATI National Platforms (as of May 2020)

EUPATI National Platforms...

bring all stakeholders together in countries address educational needs in R&D disseminate EUPATI’s training material to patient

organisations

Evolution of ENP Network21 Established ENPs

What are ENPs doing?

ENP social media campaigns

ENP learning webinars

ENP workshops & conferences

ENP MoUs with partnersENPs on the TV

ENPs in the papers

The EUPATI Toolbox on Medicines R&D

EUPATI Toolbox on Medicines R&D in 9+ languages: Huge demand

• In English, French, Italian, Spanish, German, Polish, Russian, Dutch, Danish

• >2,000,000 unique users in 1 year

• App. 3,700 000 users since launch

• Visitors from 232 countries

EUPATI Toolbox on Medicines R&D:>3.500 content items

Infographics

ArticlesPowerPoints

Fact sheets

Access via different devices

Sessionsby country

Device % of usersDesktop 70%Mobile 27%Tablet 3%

Top articles viewed on website

0 100 200 300 400 500 600 700

/hta-systems-in-europe//making-medicine-step-1-pre-discovery/

/biomarkers//marketing-authorisation/

/key-principles-pharmacology//efficacy-safety-medicines/

/clinical-trial-designs//best-practices-for-patient-involvement-…

/phases-clinical-development//an-introduction-to-clinical-research/Top articles (EN)

Site categories in order of popularity (EN)

0 200 400 600 800 1000 1200

PharmacoepidemiologyNon Clinical Studies

Benefit and Risk AssessmentSafety of Medicines

Personalised MedicineRegulatory AffairsTypes of Medicine

Pharmaceutical DevelopmentHealth Technology Assessment

Drug DiscoveryClinical Development and Trials

Basics of Medicines Development

The EUPATI Mini-CourseStarter Kits

EUPATI Mini-Course Starter Kitsto run training days and courses

Core set of PPT slides in specific area of patient involvement,plus links to EUPATI Toolbox, Case Studies + Trainers Manual!

Patient involvement in…

Research designand Planning

ResearchPriorities

Research conduct andoperations

Dissemination Communication Post-approval

Setting researchpriorities

Protocol synopsis

Protocol design

Trial Steering Committee

Information to participants

Data MonitoringCommittee

Investigator meetings

Regulatory Affairs

Fundraising for research

Practical Considerations

Patient Information

Informed Consent

Ethics Review

Study Reporting

HealthTechnology Assessment

Post-Study Communication

EUPATI Mini-Course Starter Kitsto run training days

The EUPATI Guidanceson the interaction of patients with regulators, HTA bodies,

industry and ethics committees

EUPATI Guidances for patient involvement and interaction – Why? Patients & patient organisations to be involved more widely

• include early & post-approval stages, not confined to clinical development

Overarching guidance on meaningful and ethical interaction is missing in many areas, especially in R&D, causing insecurity• Existing codes/regulations hold mostly legal & ethical

content, i.e. collaboration, communication, funding -- do not cover patient involvement in R&D

• e.g. EFPIA, EATG code, PCWP framework, ECAB, EMA framework, HTAi

Language needs to be more directive towards patient involvement • Clear default statement that collaboration is allowed unless

expressly forbidden

EUPATI Guidances to support the interaction with patient organisations in R&D

EUPATI has developed guidance documents for the interaction of patient organisations with stakeholders in industry-led R&D in HTA bodies in regulatory processes in ethics committees

Available for download at EUPATI.eu > Resources > Guidances

The EUPATI webinar library

EUPATI branded and co-branded webinars EUPATI has been producing a series of webinars on key

aspects of the medicine R&D and clinical development process Most webinars produced by EUPATI team and alumni Co-production with and technical support to patient groups for

their webinars All content free and publicly accessible with slide decks

attached

https://www.eupati.eu/category/webinar/

The EUPATI Production and Quality Assurance Process

Solid EUPATI production & review process to guarantee objectivity

In consultation of the external advisory boards, EUPATI has implemented a solid multi-stage multi-stakeholder production and review process

Internal production and review process• Content created by experts from different stakeholders • EUPATI Editorial Group (comprising patient organisations, academia,

Industry, NGOs) collectively reviewed each topic to ensure content is consistent, avoids unnecessary duplication and is fit for intended audience/purpose.

External testing and review process• User-tested with target audience• Reviewed by independent Project Advisory Board members (e.g.

journalists, academics, ethics committee members)• Relevant topics reviewed by independent regulatory experts and HTA

experts• edited for appropriate language by health literacy experts

Solid EUPATI production & review process to guarantee objectivity

What will EUPATIdo next?

Please be in touch!info@eupati.eu